Assessment of a PERT-free Nutritional Therapeutic Delivery System for Cystic Fibrosis

NCT ID: NCT04966897

Last Updated: 2021-07-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-08-31
• Study Completion Date: 2019-09-30

Brief Summary

This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.

Detailed Description

This is a single center, randomized, double-blind, cross-over trial (with a follow-on single-blind safety evaluation stage), assessing a ready-to-drink nutritional supplement used without PERT ("PERT-free"), nutritional supplement for blood lipid levels, safety and tolerability compared to a standard of care nutritional supplement used concomitantly with PERT.

The study hypothesis is that the PERT-free nutritional supplement will be equivalent to, or superior to, a standardized nutritional shake with regard to fat absorption, as indicated by blood lipid (triglyceride) levels while maintaining tolerability and safety without the use of PERT. The objective is to evaluate efficacy, safety, and tolerability of a "PERT-free" nutritional shake compared to a standard nutritional shake used concomitantly with PERT. Measurements include components of a standard lipid blood panel (triglycerides, cholesterol, HDL cholesterol, LDL cholesterol, and VLDL cholesterol), absorption of fat-soluble vitamins (vitamin D) and symptoms associated with EPI. Other objectives include evaluating palatability and patients' impressions of the products.

Conditions

Cystic Fibrosis; Exocrine Pancreatic Insufficiency

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

GBNS + PERT placebo

GBNS + PERT placebo (drink volume sufficient to supply 0.5 g of MAG per kg of body weight plus PERT placebo capsules according to patient body weight.

Group Type: EXPERIMENTAL

GlycosBio Nutritional Supplement + PERT placebo

Intervention Type: DIETARY_SUPPLEMENT

The lipid (fat) in the GlycosBio Nutritional Supplement (GBNS) was provided in the form of a re-structured lipid monoacylglyceride (MAG) produced by the study sponsor. MAGs are readily absorbed by enterocytes without the need of digestion by pancreatic lipases. The "MAG oil" is produced enzymatically from almond oil. The product also contains essential amino acids, carbohydrates as simple sugars and fat-soluble vitamins and minerals. The GBNS to be administered in a volume sufficient to supply 0.5 g of MAG per kg of body weight

Standard Nutritional Supplement + PERT

Standard nutritional supplement + PERT (drink volume sufficient to supply 0.5 g of TAG per kg of body weight plus PERT capsules according to body weight).

Group Type: ACTIVE_COMPARATOR

Standard Nutritional Supplement + PERT

Intervention Type: DIETARY_SUPPLEMENT

A 5.9% fat triacylglycerol-based commercially available nutritional supplement (Boost Plus®, Nestlé Health Science, Bridgewater, NJ) in a volume sufficient to supply 0.5 g of TAG per kg of body weight over the same time period and PERT capsules (24,000 iu/capsule) at a dose of 2,500 iu of lipase activity per gram of fat ingested.

Interventions

GlycosBio Nutritional Supplement + PERT placebo

The lipid (fat) in the GlycosBio Nutritional Supplement (GBNS) was provided in the form of a re-structured lipid monoacylglyceride (MAG) produced by the study sponsor. MAGs are readily absorbed by enterocytes without the need of digestion by pancreatic lipases. The "MAG oil" is produced enzymatically from almond oil. The product also contains essential amino acids, carbohydrates as simple sugars and fat-soluble vitamins and minerals. The GBNS to be administered in a volume sufficient to supply 0.5 g of MAG per kg of body weight

Intervention Type: DIETARY_SUPPLEMENT

Standard Nutritional Supplement + PERT

A 5.9% fat triacylglycerol-based commercially available nutritional supplement (Boost Plus®, Nestlé Health Science, Bridgewater, NJ) in a volume sufficient to supply 0.5 g of TAG per kg of body weight over the same time period and PERT capsules (24,000 iu/capsule) at a dose of 2,500 iu of lipase activity per gram of fat ingested.

Intervention Type: DIETARY_SUPPLEMENT

Eligibility Criteria

Inclusion Criteria

• Provision of signed and dated informed consent form.
• Stated willingness to comply with all study procedures and availability for the duration of the study.
• Male or female, aged 12 years or older.
• Diagnosis of cystic fibrosis
• Currently receiving treatment with a commercially available pancreatic enzyme product for more than 3 months.
• Clinically stable condition without evidence of acute respiratory disease within 1 month of enrollment.
• Stable body weight defined as no more than 5% decline within 3 months of enrollment.
• Females of child-bearing potential should agree to continue using a medically acceptable method of birth control throughout the study and for 30 days immediately after the last dose of study drug. Medically acceptable methods of birth control include bilateral tubal ligation or the use of either a contraceptive implant, a contraceptive injection (Depo-Provera™), an intrauterine device, or an oral contraceptive taken within the past 3 months where the subject agrees to continue using during the study or to adopt another birth control method, or a double-barrier method which consists of a combination of any two of the following: diaphragm, cervical cap, condom, or spermicide.
• Ability to take oral medication and oral liquid nutritional supplements and be willing to adhere to the study interventions.
• Agreement to adhere to Lifestyle Considerations as defined by the study protocol throughout the duration of the study.

Exclusion Criteria

• Evidence of cardiovascular, respiratory (except underlying disease), urogenital, gastrointestinal/hepatic (except underlying disease), hematologic/immunologic, head, ears, eyes, nose, throat, dermatologic/connective tissue, musculoskeletal, metabolic/nutritional (except underlying disease), endocrine (except controlled diabetes mellitus), neurologic/psychiatric, milk, nut or soy allergies, recent major surgery, or other relevant diseases as revealed by history, physical examination and/or laboratory assessments, which could limit participation in or completion of the study.
• History of acute abdomen in the last year.
• History of fibrosing colonopathy.
• History of distal intestinal obstruction syndrome (DIOS) within 6 months prior to enrollment.
• Solid organ transplant or surgery affecting the large bowel other than appendectomy.
• Small bowel surgery that significantly affected absorptive capacity (e.g. gastrectomy or pancreatectomy).
• Intestinal inflammatory diseases including chronic diarrheal illness unrelated to pancreatic insufficiency.
• Celiac disease or Crohn's disease.
• Receiving enteral tube feeds for ≥50% of daily calorie intake.
• Pregnancy or lactation.
• Any type of malignancy involving the digestive tract in the last 5 years.
• Known allergy to pancreatin or inactive ingredients (excipients) of pancreatin capsules.
• Suspected non-compliance or non-cooperation.
• Intake of experimental drugs within 30 days prior to study start.
• Mental disability or any other lack of fitness, in the Investigator's opinion, to preclude subject's participation in or ability to complete the study.
• Diagnosis of human immunodeficiency virus in medical history.
• Listed for lung transplantation or other solid organ transplant or documented forced expiratory volume (FEV) ≤25%.
• Use of lipid lowering therapy including statins, fibrates, niacin, and proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors that cannot be held at least 14 days prior to Day 1 and through Day 15 of the study.

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baylor College of Medicine

OTHER

Sponsor Role: collaborator

GlycosBio, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Clinical Research Center (CRC) at Texas Children's Hospital

Houston, Texas, United States

Countries

United States

Other Identifiers

GLYCOS-001-CF17

Identifier Type: -

Identifier Source: org_study_id